Stoke Therapeutics, Inc.

Stoke Therapeutics, Inc. is a biotechnology company dedicated to developing RNA-based medicines to address the underlying causes of severe genetic diseases by precisely upregulating protein expression. Founded in 2014 and headquartered in Bedford, Massachusetts, with additional offices in Cambridge, Massachusetts, the company aims to restore target proteins to near normal levels.

The company utilizes its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to design antisense oligonucleotides (ASOs) that selectively restore protein levels. Its lead clinical candidate, zorevunersen (STK-001), is currently in Phase 3 clinical trials for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke's pipeline also includes STK-002 for Autosomal Dominant Optic Atrophy (ADOA), which is in Phase 1 clinical trials or preclinical stages, and earlier-stage programs for SYNGAP-1 disorders and Rett syndrome. The company's initial focus is on haploinsufficiency diseases of the central nervous system and the eye.

Stoke Therapeutics is a publicly traded company on NASDAQ under the ticker symbol STOK. Ian F. Smith serves as the Chief Executive Officer, having been appointed in October 2025. Recent developments include the appointment of Clare Kahn, Ph.D., to its Board of Directors in April 2026. The company anticipates completing enrollment for its Phase 3 EMPEROR study for zorevunersen in Q2 2026, with a data readout expected in mid-2027 and a rolling New Drug Application (NDA) submission planned for the first half of 2027 under Breakthrough Therapy designation. Stoke has also established collaborations with companies like Biogen and Acadia Pharmaceuticals.

Latest updates

Stoke Therapeutics to Detail Q2026 Progress Amidst Dravet Syndrome Trial

Event summary

  • Stoke Therapeutics (STOK) will host a webcast and conference call on May 7, 2026, at 4:30 PM ET to discuss Q2026 business and financial updates.
  • The company's lead investigational medicine, zorevunersen, is in Phase 3 development for Dravet syndrome.
  • Zorevunersen has received FDA orphan drug, rare pediatric disease, and Breakthrough Therapy designations.
  • Stoke has a strategic collaboration with Biogen for the development and commercialization of zorevunersen, with Biogen holding rest-of-world rights.

The big picture

Stoke's progress hinges on the success of zorevunersen, a first-in-class treatment for Dravet syndrome, a rare and debilitating neurological disorder. The company's TANGO platform represents a novel approach to RNA medicine, but its broader applicability remains to be proven. The partnership with Biogen provides Stoke with valuable resources and expertise, but also introduces complexities related to commercialization and intellectual property.

What we're watching

Clinical Outcomes
The Phase 3 trial data for zorevunersen will be critical; any signs of efficacy or safety concerns will significantly impact Stoke's valuation and Biogen's commercialization strategy.
Commercialization
The ongoing collaboration with Biogen will be under scrutiny, as the split of commercialization rights creates potential for future disputes or renegotiations.
Pipeline Expansion
The company’s ability to expand its TANGO platform beyond Dravet syndrome and its initial CNS/eye focus will determine its long-term growth potential.
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